<code id='8DB64099F5'></code><style id='8DB64099F5'></style>
    • <acronym id='8DB64099F5'></acronym>
      <center id='8DB64099F5'><center id='8DB64099F5'><tfoot id='8DB64099F5'></tfoot></center><abbr id='8DB64099F5'><dir id='8DB64099F5'><tfoot id='8DB64099F5'></tfoot><noframes id='8DB64099F5'>

    • <optgroup id='8DB64099F5'><strike id='8DB64099F5'><sup id='8DB64099F5'></sup></strike><code id='8DB64099F5'></code></optgroup>
        1. <b id='8DB64099F5'><label id='8DB64099F5'><select id='8DB64099F5'><dt id='8DB64099F5'><span id='8DB64099F5'></span></dt></select></label></b><u id='8DB64099F5'></u>
          <i id='8DB64099F5'><strike id='8DB64099F5'><tt id='8DB64099F5'><pre id='8DB64099F5'></pre></tt></strike></i>

          explore

          explore

          author:explore    Page View:847
          Gene therapy helped Conner Curran, right, walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. -- Biotech coverage from STAT
          Gene therapy helped Conner Curran (at right) walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. Courtesy Jess & Chris Curran

          In the beginning, Conner Curran ran up the stairs.

          It was a miraculous moment for him and his parents, Jess and Chris Curran. Just months before, contractors were in their New York home to install a motorized lift, while Jess tearfully explained that it wasn’t for a grandparent but for their 7-year-old son, who suffered from Duchenne muscular dystrophy and could barely haul himself up the steps.

          advertisement

          Then Conner was accepted as the first patient in Pfizer’s Duchenne gene therapy trial. Three weeks later, he could bound to the second floor. Soon, he could walk the two-plus miles to Goldberg’s, a local bagel shop. “I couldn’t do that before,” he told NPR in 2020. 

          Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

          GET STARTED Log In

          focus

          Trump demands the U.S. pay no more for drugs than other countries … again
          Trump demands the U.S. pay no more for drugs than other countries … again

          EthanMiller/GettyImagesWASHINGTON—FormerPresidentTrumpisbacktocampaigningfortyingMedicaredrugpricest

          read more
          HIV vaccine remains elusive. Immunologists keep trying new ideas
          HIV vaccine remains elusive. Immunologists keep trying new ideas

          SethPincus,ElizabethFischerandAustinAthman,NationalInstituteofAllergyandInfectiousDiseases/NIHHIVhas

          read more
          Activists slam private health insurers over coverage restrictions
          Activists slam private health insurers over coverage restrictions

          ProtestorsoutsideAmerica'sHealthInsurancePlansheadquarters.CourtesyPeople'sActionWASHINGTON—Hun

          read more

          JPM 2024: FogPharma’s CEO unveils ‘contrarian’ plan in oncology

          FogPharmaCEOMathaiMammenVenrockSANFRANCISCO—SixmonthsintohistenureasCEOofcancerstartupFogPharma,Math